RARE BEST PRACTICES COURSE

ROMA (Feb 23-24 2015)

Euro-HSPMadrid

2015 May 30

Jean Bénard

May 31, GA 2015 Madrid

Deals with RD as a global health issue,Deals with RD as a global health issue,

Exploiting and integrating contributions from all EU Exploiting and integrating contributions from all EU

Members and other world areas (Caucasus, Europe, Members and other world areas (Caucasus, Europe,

America, Oceania, PAHO/WHO)America, Oceania, PAHO/WHO)

Will identify additional research needs to further improve Will identify additional research needs to further improve

clinical practice.clinical practice.

Euro-HSP RARE BEST PRACTICES( RBP) RARE BEST PRACTICES( RBP)

Platform Platform

Aims at improving clinical management of rare disease patients Aims at improving clinical management of rare disease patients and narrowing the existing gap in quality of health care among and narrowing the existing gap in quality of health care among EU member states as well as in other countries. EU member states as well as in other countries.

This will be achieved by collecting, evaluating and This will be achieved by collecting, evaluating and disseminating best practices as well as sharing knowledge. disseminating best practices as well as sharing knowledge.

The ultimate impact of the RARE-Bestpractices project should The ultimate impact of the RARE-Bestpractices project should be an improvement of health outcomes and quality of life for be an improvement of health outcomes and quality of life for rare diseases patients, through a reduction of inequalities in rare diseases patients, through a reduction of inequalities in health care and an increased equity of access to better health care and an increased equity of access to better diagnostics and therapeutics at EU level.diagnostics and therapeutics at EU level.

Euro-HSP RARE BEST PRACTICE (RBP) RARE BEST PRACTICE (RBP)

ProjectProject

Document with recommendations, aimed to improve the quality of Document with recommendations, aimed to improve the quality of

health, based on systematic reviews of scientific studies combined health, based on systematic reviews of scientific studies combined

with a consideration of harms and benefits of different care optionswith a consideration of harms and benefits of different care options

Supplemented with expertise and experience from care providers Supplemented with expertise and experience from care providers

and care users.and care users.

Dutch guideline for guidelines, 2012, based on IOMDutch guideline for guidelines, 2012, based on IOM

Euro-HSP RARE BEST PRACTICE (RBP) RARE BEST PRACTICE (RBP)

GuidelineGuideline

An algorithm integrating numerous criteria has been built that yields a chek-list enabling to appraise a document proposed as a guideline...........................................................................................................................Domain 1: Scope and Purpose....................................................................................................Domain 2: Stakeholder Involvement..........................................................................................Domain 3: Rigour of Development.............................................................................................Domain 4.Clarity of Presentation...............................................................................................Domain 5.Applicability...............................................................................................................Domain 6: Editorial Independence........................................................................................... Overall Guideline Assessment

RARE BEST PRACTICE (RBP)RARE BEST PRACTICE (RBP)Agree II InstrumentAgree II Instrument

EFNS guidelines on the Molecular Diagnosis EFNS guidelines on the Molecular Diagnosis of Ataxias and Spastic Paraplegias of Ataxias and Spastic Paraplegias

T. Gassera T. Gassera et alet al. . European Journal of NeurologyEuropean Journal of Neurology 2010, 17: 2010, 17: 179–188 179–188

based on the expertise of Physiotherapists of all Europe: based on the expertise of Physiotherapists of all Europe: Austria, Belgium, France, Hungary, Israël, Italy, Norway, Austria, Belgium, France, Hungary, Israël, Italy, Norway, UK, SwitzerlandUK, Switzerland

RARE BEST PRACTICE (RBP)

This guidance is targeted at paediatric physiotherapists involved in the This guidance is targeted at paediatric physiotherapists involved in the management of children with neurological conditions where botulinum management of children with neurological conditions where botulinum toxin (BTX-A) may be an appropriate adjunctive treatment.toxin (BTX-A) may be an appropriate adjunctive treatment.

To date there are no standardised pathways or consensus of practice To date there are no standardised pathways or consensus of practice for providing BTX-A injection services for children. Current provision and for providing BTX-A injection services for children. Current provision and practice varies from one locality to another throughout the UK.practice varies from one locality to another throughout the UK.

Nationally, paediatric physiotherapists have expressed a need for Nationally, paediatric physiotherapists have expressed a need for evidence/consensus-based guidance as there is littleevidence/consensus-based guidance as there is littlespecific advice regarding physiotherapy intervention following BTX-A specific advice regarding physiotherapy intervention following BTX-A treatment.treatment.

EVIDENCE-BASED GUIDANCE FOR EVIDENCE-BASED GUIDANCE FOR PHYSIOTHERAPISTSPHYSIOTHERAPISTS

The use of Botulinum Toxin in Children with The use of Botulinum Toxin in Children with Neurological ConditionsNeurological Conditions

RARE BEST PRACTICE (RBP)RARE BEST PRACTICE (RBP)